The US Food and Drug Administration (FDA) has granted orphan drug designation for the novel agent known as TRC105, in development by Tracon Pharmaceuticals for the treatment of soft-tissue sarcoma.
Orphan drug status can grant the company tax credits and a reduction in certain fees associated with drug development and approval, in an effort to aid the development of agents created to address diseases affecting fewer than 200,000 people in the United States. About 13,000 new cases of soft-tissue sarcoma are diagnosed each year.
TRC105 is a novel antibody to endoglin, a protein that is overexpressed in endothelial cells and that is essential for angiogenesis.
“Orphan drug designation for TRC105 underscores the high level of unmet medical need in patients with soft-tissue sarcomas and validates our commitment to developing TRC105 in a variety of orphan drug indications,” said Charles Theuer, MD, PhD, the CEO of Tracon, in a press release. “We are highly encouraged by the clinical data reported to date from the phase Ib/II trial of TRC105 in sarcoma patients, especially in patients with angiosarcoma. We look forward to the availability of complete results from the phase II portion of the sarcoma trial and initiating a pivotal phase III trial in patients with angiosarcoma in 2016.”
The phase Ib/II trial has an estimated enrollment of 94 patients, with an estimated completion date of December 2016. It is testing TRC105 in combination with pazopanib for patients with unresectable soft-tissue sarcoma. The company also has ongoing clinical trials of the drug in advanced renal cell carcinoma, hepatocellular carcinoma, non–squamous cell lung cancer, and breast cancer.
Though sarcoma patients still represent an underserved population, there has been some recent progress in the field. In January, the FDA approved eribulin for the treatment of unresectable, metastatic liposarcoma. It was the first sarcoma agent to show a significant improvement in survival time.
The agency also approved trabectedin for the treatment of liposarcoma and leiomyosarcoma in October 2015, and granted orphan drug status to GPX-150, an analog of doxorubicin in testing for patients with soft-tissue sarcoma, in January of this year.